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COMPOUND, PHARMACEUTICAL COMPOSITION and METHOD
专利权人:
FONDAZIONE TELETHON
发明人:
Andrea Ballabio,Giancarlo Parenti
申请号:
BR112015008673
公开号:
BR112015008673A2
申请日:
2013.10.17
申请国别(地区):
BR
年份:
2017
代理人:
摘要:
compound, pharmaceutical composition and abstract method the present invention relates to a transcription factor eb (tfeb) protein, ortholog, recombinant or synthetic or biotechnological functional derivative thereof, allelic variant thereof and fragments thereof; a chimeric molecule comprising the teb protein, ortholog, recombinant or synthetic or biotechnological functional derivative thereof, allelic variant thereof and fragments thereof; a polynucleotide coding for said protein or ortholog, recombinant or synthetic or biotechnological functional derivative thereof, allelic variant thereof and fragments thereof; the comprising said polynucleotide vector; a host cell genetically engineered expressing said polypeptide or a pharmaceutical composition for use in the treatment or prevention of a glycogen storage disease. preferably of pompe or danon disease.composto, composição farmacêutica e método abstract the present invention relates to a transcription factor eb (tfeb) protein, ortholog, recombinant or synthetic or biotechnological functional derivative thereof, allelic variant thereof and fragments thereof; a chimeric molecule comprising the tfeb protein, ortholog, recombinant or synthetic or biotechnological functional derivative thereof, allelic variant thereof and fragments thereof; a polynucleotide coding for said protein or ortholog, recombinant or synthetic or biotechnological functional derivative thereof, allelic variant thereof and fragments thereof; a vector comprising said polynucleotide; a host cell genetically engineered expressing said polypeptide or a pharmaceutical composition for use in the treatment or/and prevention of a glycogen storage disease. preferably of pompe or danon disease.
来源网站:
中国工程科技知识中心
来源网址:
http://www.ckcest.cn/home/

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